Hartmann Wellhoefer, MD, Vice President, Medical Affairs, Rare Disease and Internal Medicine, Global R&D at Takeda sat down with CheckRare to discuss the 15th Annual WORLDSymposium 2019 to share his insights about this year’s conference including key highlights and trends and Takeda’s presence.
Dr. Wellhoefer, can you tell us a what makes the WORLDSymposium a meaningful and unique conference?
The annual WORLDSymposium for lysosomal storage disorders (LSD) is the only dedicated conference where patient advocacy organizations, clinicians and representatives from industry and academia come together to focus on the latest discoveries and clinical advances in LSDs. It’s also unique because it is smaller and more focused than many conferences, allowing for greater interaction for the LSD community and discussion of needs – particularly patient needs – of those affected by LSDs.
What were some of the major trends that were highlighted at the WORLDSymposium this year?
The WORLD meeting this year covered a variety of exciting programs and highlighted scientific and clinical progress being made in different lysosomal storage disorders, including those areas where we at Takeda are focused: Hunter syndrome (also known as Mucopolysaccharidosis II or MPS II), Gaucher disease, Fabry disease and Metachromatic Leukodystrophy (also known as MLD).
The major trend and topic of excitement at WORLD was gene therapy and the role it hopefully will play in LSDs. While gene therapy is not new, it was a noticeable topic of discussion this year, with a real buzz about new scientific data and more attempts to bring gene therapy to LSDs. There was a lot of talk about the potential of gene therapy to treat LSDs and we are also active in this space. However, it is still an open question when gene therapy can be brought to patients with LSDs outside clinical trials. The hurdles gene therapy is facing are still high, yet there seems to be increasing optimism about the potential for this category for this group of patients.
Another major topic at WORLD was early diagnosis and newborn screening for rare genetic diseases such as LSDs, including discussions on how newborn screening could bring existing or new treatment to patients earlier. In contrast to gene therapy, early diagnosis and newborn screening is right around the corner in the U.S., and in some cases already here. There are state-wide newborn screening programs in the U.S. and more countries are also seriously considering an expansion of these newborn screenings of lysosomal storage disorders.
Why was it important for Takeda to attend this meeting?
We had a large presence at WORLD this year with 12 presentations, including 11 posters and one oral presentation, as well as a satellite symposium on biomarkers in the clinical management of LSDs, and a large booth in the exhibit hall. Through this strong presence at the WORLD, we wanted to demonstrate Takeda’s commitment to supporting the LSD community, including what we are doing to advance potential treatments in this area of high unmet need. WORLD also allowed us to reconnect and engage with the world’s leading LSD experts, clinicians and patient organizations, who all share our passion for supporting patients affected by rare disease, particularly lysosomal storage disorders.
Can you provide more specific clinical highlights that Takeda presented at WORLD?
As I mentioned, we had a lot of activities at WORLD. We held a symposium on biomarkers in lysosomal disorders called: “Biomarkers: Ready for Prime Time in Clinical Management”, that was extremely well attended. In fact, the room was filled to the point where some people had to sit on the floor as there were no chairs left. This interest reflects the importance of biomarkers in lysosomal storage disorders.
As part of the 12 posters and presentations – a testament to our research efforts in this space – there are three areas I would like to highlight in particular. Firstly, we presented data on our Patient-Reported Outcomes Tool for Gaucher disease. This tool was developed in collaboration with clinicians and patient advocacy organizations. The patient-reported outcomes data we presented at WORLD are very important and reflect our patient-centric approach: how we try to better understand what benefits are important to patients and how their needs can be addressed and demonstrated in the context of a clinical trial.
Another area that is consistent with our patient-centric approach and illustrates our commitment to rare diseases is some of the evidence we presented for Fabry disease. We showed data that evaluated the impact on renal function with enzyme replacement therapy over a 12-year time frame. Some of the patients in this data set were followed up for 18 years, which is extremely long from a rare disease population perspective.
Thirdly, Takeda is a science- and innovation-driven company. We were very proud to present the Phase 2b clinical trial design for an investigational compound being evaluated for Metachromatic Leukodystrophy (MLD), a study we are looking to move into patient enrollment in the not-so-distant-future. We sparked significant interest in this MLD trial, a testament to the huge unmet need in MLD.
In closing, do you have any last thoughts you would like to provide?
At Takeda, we are committed to doing more to help improve people’s lives. We are driven by our passion to realize this goal by providing innovative medicines. As a rare disease leader, we stand with millions of patients, families, caregivers and healthcare professionals affected by rare diseases, championing them as we continue our pursuit of better health through innovative treatments. At WORLD, I hope we were able to demonstrate our commitment to the lysosomal storage disorder community. The opportunity to interact there with the patient organizations provided us with profound insights that help us better understand the diseases and to better address the needs of the patients and families affected by them.
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